March 20th, 2014. Agenda:. Potential elimination of the Orphan Drug Tax Credit - Joel White & Jill Schmalz , Horizon Government Affairs Community efforts to encourage CMS to revise its proposed rule on Medicaid “line extension” rebates – Jennifer Bernstein , Horizon Government Affairs
Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author.While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server.
Joel White, Jen Bernstein and Jill Schmalz
Horizon Government Affairs
March 20, 2014
Joel White Jen Bernstein Jill Schmalz
President Executive Vice President Senior Vice President
(202) 559-0192 (202) 559-0197 (202) 803-6031
Would exclude ALL pharmaceuticals indicated by the Food and Drug Administration solely for the treatment of a rare disease from the branded prescription drug fee imposed under the Patient Protection and Affordable Care Act.
….for those you love
Brief Background on Cal:
In 2006, a young boy by the name of Cal passed away when he was five years due to an undiagnosed disease. During his short life, Cal developed many unexplained medical problems such as neurological difficulties, cerebral atrophy, migraines, and seizures. Doctors handling Cal’s case could only tell his parents what diseases he did not have, and tragically Cal’s parents never received a confirmed diagnosis.
If there had been a medical database, where symptoms of rare diseases could be shared; doctors could have collaborated on Cal’s case and confirmed a diagnosis.
Currently, there is no database like this that is available, and the only way to compare cases is by word of mouth.
Facts about Undiagnosed Diseases:
According to the Department of Health and Human, undiagnosed diseases are considered to be disorders with long-standing symptoms or signs that have not been diagnosed; despite extensive clinical investigation.
Undiagnosed diseases include rare diseases that less than 200,000 individuals in the US have, yet-to-be-described disorders, or rare variants of more common diseases.
According to the European Organization for Rare Disease, 25% of diagnoses are delayed between 5-30 years. This delay in an accurate diagnosis can be costly by involving numerous tests, medications, therapies, travel, and special diets. These expenses can leave the patients and loved ones emotionally and physically drained.